Frequently Asked Questions

1
What is the purpose of an Expanded Access Program (EAP)?

The intent of an expanded access program is to provide patients with access to investigational medication for serious diseases or conditions where there is no comparable or satisfactory alternative therapy available. The FDA has authorized the use of deflazacort* for eligible patients with DMD under the ACCESS DMDTM expanded access program. The FDA’s authorization of the ACCESS DMDTM program does not mean that the FDA has concluded that deflazacort* is safe and effective for DMD. To learn more about expanded access programs, please visit Expanded Access Program Information for Patients

2
What is the difference between an expanded access program and a clinical study?

Expanded access is a general term often used to refer to a variety of programs available to provide investigational treatment access to patients with life-threatening or severely debilitating disease for whom commercially available therapies have failed or are not available. The distinction between an expanded access program and a clinical trial is that the use of an investigational medication in an expanded access program is intended for treatment, whereas the use of an investigational medication in a clinical trial is intended to answer questions about the safety and effectiveness of the investigational medication.

3
Is everyone diagnosed with DMD eligible for this expanded access program?

No. You are encouraged to speak to your physician who treats your DMD about the possibility of taking part in the program. Alternatively, a letter can be given to your physician who treats your DMD to explain more about the program. This letter can be found in the Resources section under Patient to Physician Letter. You can also browse a list of physicians already participating in ACCESS DMDTM using the following link.

4
What is deflazacort*, the investigational medication that is being offered?

An investigational medication is a medication that is still in research and development and that has not been approved by the FDA for the proposed use under study. Deflazacort* is an investigational glucocorticoid that has not been approved by the FDA for any indication and is therefore not proven to be safe and effective. Deflazacort* is currently under investigation as a potential treatment for DMD in both pill and liquid formulations. Deflazacort* is approved for use in a wide range of conditions in Europe and Latin America, however, it is not approved for DMD in any country in the world.

5
Do I need to ask my physician who treats my DMD if I can participate in this expanded access program?

Yes. You will need to speak to your physician who treats your DMD about the possibility of taking part in this program. Your physician who treats your DMD will need to register online in order for you to participate and receive deflazacort*. Your physician’s online registration process can take a few weeks or longer. Even if your physician successfully registers, you will only be able to participate and receive deflazacort* through ACCESS DMDTM once your physician determines that you are eligible and you complete the enrollment process, which includes reviewing and signing the Informed Consent form.

6
Can I participate in other clinical studies while being in this program?

Patients participating in ACCESS DMDTM cannot participate in any other clinical studies involving deflazacort*. However, patients who are participating in clinical studies for other investigational DMD treatments/therapies may still be eligible for participation in ACCESS DMDTM. You should talk to your physician who treats your DMD to determine what is best for you.

7
If my physician who treats my DMD feels I am eligible, but does not want to participate, is there a way for me to still take part in the program?

Yes. You and your physician who treats your DMD can review the list of participating physicians in your area and decide if it is a good option for you to visit a physician who treats DMD and who is currently participating in ACCESS DMDTM.

8
If I am currently taking a glucocorticoid such as prednisone or deflazacort* can I still participate?

You may be able to participate in ACCESS DMDTM. You should talk to your physician who treats your DMD to determine what is best for you.

9
My next appointment is several months away. Can I sign up now or do I have to wait until my next appointment?

You should talk to your physician who treats your DMD to determine what is best for you.

10
What will I be asked to do if I take part in this program?

If you agree to enter this program, your physician who treats your DMD may ask you to visit their clinic to confirm you are eligible for participation. You will be required to sign a consent form to begin participation. Your physician who treats your DMD will then decide if you should take deflazacort*, and if so, whether in pill or liquid form. You can talk to your physician who treats your DMD about this process.

11
How long will I take part in the program?

If you participate in ACCESS DMDTM, you may continue participating until one of the following occurs: (a) deflazacort* is approved in the U.S. (if approved) and made commercially available; (b) the ACCESS DMDTM program is terminated; (c) your physician who treats your DMD determines that you should stop taking part in the program; or (d) you decide you no longer want to participate.

12
How will I be compensated for my participation in this program?

While participating in ACCESS DMDTM, deflazacort* will be provided to participants at no cost. You will not be paid to take part in ACCESS DMDTM.

13
What is the role of the specialty pharmacy in this program?

A specialty pharmacy is different from traditional pharmacies in that it helps coordinate many aspects of patient care and disease management. For the purposes of this program, the specialty pharmacy will be involved in deflazacort* delivery and return.

14
Who will have access to my information collected during participation?

Any personally identifiable information (for example, contact details required for shipping and distributing the investigational medication) will not be accessible to anyone who is not directly associated with ACCESS DMDTM, except with the patient or caregiver’s permission or as required by law.

Any information that is gained from this expanded access program may be used for publishing outcomes and for regulatory purposes by the sponsor, Marathon Pharmaceuticals LLC. However, this information will be combined with other participant data and will not identify any individual participant.

15
Who can I speak to if I still have questions?

You should talk to your physician who treats your DMD if you have questions about this program. Alternatively, you may contact +1-844-800-4DMD (4363).

*Deflazacort is an investigational medication that has not been approved by the Food and Drug Administration (FDA) and is therefore not proven to be safe and effective.